Rett SyndromePhase 3NCT04181723
Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™)
A Phase 3 study testing Trofinetide in people with Rett Syndrome, run by ACADIA Pharmaceuticals Inc. at 21 sites in United States.
Trial summary
To investigate the efficacy of treatment with oral trofinetide versus placebo in females with Rett syndrome Read more about this study
- Who is it for?
- Ages 5 to 20 years, women only
- Conditions the study is for
- Rett Syndrome
- What is being tested?
- Trofinetide
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 187 participants
- Where
- 21 sitesUnited States
- Study dates
- 6 Nov 2019to 28 Oct 2021
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 20 years
- Women only
- No healthy volunteers
- Child, Adult
Who may be able to join
- Female subjects 5 to 20 years of age, inclusive, at Screening
- Body weight ≥12 kg at Screening
- Can swallow the study medication provided as a liquid solution or can take it by gastrostomy tube
- Has classic/typical Rett syndrome (RTT)
- Has a documented disease-causing mutation in the MECP2 gene
- Has a stable pattern of seizures, or has had no seizures, within 8 weeks of Screening
Show all 9Show fewer
- Subjects of childbearing potential must abstain from sexual activity for the duration of the study and for at least 30 days thereafter or must agree to use acceptable methods of contraception. Subject must not be pregnant or breastfeeding.
- The subject's caregiver is English-speaking and has sufficient language skills to complete the caregiver assessments
- Subject and caregiver(s) must reside at a location to which study drug can be delivered and have been at their present residence for at least 3 months prior to Screening
Who cannot join
- Has been treated with insulin within 12 weeks of Baseline
- Has current clinically significant cardiovascular, endocrine (such as hypo- or hyperthyroidism, Type 1 diabetes mellitus, or uncontrolled Type 2 diabetes mellitus), renal, hepatic, respiratory or gastrointestinal disease (such as celiac disease or inflammatory bowel disease) or has major surgery planned during the study
- Has a history of, or current, cerebrovascular disease or brain trauma
- Has significant, uncorrected visual or uncorrected hearing impairment
- Has a history of, or current, malignancy
- Has a known history or symptoms of long QT syndrome
- Additional inclusion/exclusion criteria apply. Patients will be evaluated at screening to ensure that all criteria for study participation are met. Patients may be excluded from the study based on these assessments (and specifically, if it is determined that their baseline health and condition do not meet all pre-specified entry criteria).
Trial locations and contacts
21 sites in 1 country.
21 sites in 1 country
United States21 sites
Emory Genetics Clinical Trial Center
Atlanta, Georgia, 30322
Children's Hospital Colorado
Aurora, Colorado, 80042
Kennedy Krieger Institute - Clinical Trials Unit
Baltimore, Maryland, 21205
University of Alabama at Birmingham
Birmingham, Alabama, 35233
Boston Children's Hospital Harvard Medical School
Boston, Massachusetts, 02115
The University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599
Rush University Children's Hospital
Chicago, Illinois, 60612
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Cleveland Clinic
Cleveland, Ohio, 44195
Greenwood Genetic Center
Greenwood, South Carolina, 29646
Texas Children's Hospital
Houston, Texas, 77030
University of California, San Diego
La Jolla, California, 92093
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Translational Gemomics Research Institute (TGen)
Phoenix, Arizona, 85012
UC Davis MIND Institute
Sacramento, California, 95817
Gillette Children's Specialty Healthcare
Saint Paul, Minnesota, 55101
Seattle Children's
Seattle, Washington, 98105
Washington University School of Medicine
St Louis, Missouri, 63110
Children Medical Services
Tampa, Florida, 33606
Montefiore Medical Center, Children's Hospital at Montefiore
The Bronx, New York, 10467
About this study
Official title: A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome
To investigate the efficacy of treatment with oral trofinetide versus placebo in females with Rett syndrome
- Conditions
- Rett Syndrome
- Being tested
- Trofinetide, Placebo
- Medical subject headings
- Rett Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TrofinetideDrug
Trofinetide solution administered based on the subject's weight at Baseline, twice daily for 12 weeks
PlaceboOther
Trofinetide placebo solution administered based on the subject's weight at Baseline, twice daily for 12 weeks
| Study group | Role | What they receive |
|---|---|---|
| Drug - Trofinetide | Experimental | Trofinetide solution of 30-60 mL based on the subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube) Receives: Drug: Trofinetide |
| Placebo | Placebo comparator | Trofinetide placebo solution of 30-60 mL based on the subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube) Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score - Change From Baseline to Week 12
The RSBQ is a 45-item caregiver-completed rating scale that includes 45 items, 39 of them grouped into 8 subscales, whose ratings reflect the severity and frequency of symptoms. Items are rated as 0 (not true), 1 (somewhat or sometimes true), or 2 (very true). The 8 subscales are general mood, breathing problems, hand behavior, face movements, body rocking/expressionless face, night-time behaviors, fear/anxiety, and walking/standing. Scores for item 31 are reversed in the calculation of the total score. The total score ranges from 0 to 90 and is calculated as the sum of the item scores. Higher scores mean worse behaviour.
Measured: Baseline and Week 12
Clinical Global Impression-Improvement (CGI-I) Score at Week 12
To rate how much the subject's illness has improved or worsened relative to a baseline state, a 7-point scale is used from 1=very much improved, 2=much improved, 3=minimally improved, 4=no change, 5=minimally worse, 6=much worse, 7=very much worse.
Measured: 12 Weeks Treatment Duration
Secondary outcomes (9)
Change From Baseline to Week 12 in Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist - Social Composite Score (CSBS-DP-IT Social)
Standardized screening scale for assessing communication and pre-linguistic skills in young children 12-24 months and can be used with older children with developmental delay. The CSBS-DP includes a suite of three separate measures: The Infant-Toddler Checklist, a follow-up Caregiver Questionnaire and a Behavior Sample. In this study only the Infant-Toddler (CSBS-DP-IT) Checklist was used. The CSBS-DP-IT Checklist is a 24-item rating scale and each item is scored using a three-level rating of frequency: "not yet", "sometimes" and "often". The CSBS-DP-IT Social Composite score the range was 0 to 26 and a higher score represented a worse outcome. Three composite scores can be calculated: 1) Social Composite; 2) Speech Composite; 3) Symbolic Composite.
Measured: 12 Weeks Treatment Duration
Change From Baseline to Week 12 in Impact of Childhood Neurologic Disability Scale (ICND) Total Score
The Impact of Childhood Neurologic Disability (ICND) scale evaluates the effect of four conditions or health problems on 11 aspects of a child's or family's life as "A lot", "Some", "A little", "Not at all", or "Does not apply". The four conditions or health problems are 1) inattentiveness, impulsivity, or mood, 2) ability to think and remember, 3) neurologic or physical limitations, and 4) epilepsy. The ICND score the range of 0 to 132 and a higher score represents a worse outcome.
Measured: 12 Weeks Treatment Duration
Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Hand Function (RTT-HF)
Clinical assessment of the subject's ability to use their hands for functional purposes (such as reaching for and grasping objects, self-feeding, or drawing). The assessment is made on an 8-point Likert scale (0-7) with 0 denoting normal functioning and 7 the most severe impairment.
Measured: 12 Weeks Treatment Duration
Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ambulation and Gross Motor Skills (RTT-AMB)
Clinical assessment of the subject's ability to sit, stand, and ambulate (e.g., walking, running, and climbing stairs). The assessment was made on an 8-point Likert scale (0 to 7), with 0 denoting normal functioning and 7 as the most severe impairment.
Measured: 12 Weeks Treatment Duration
Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ability to Communicate Choices (RTT-COMC)
Clinical assessment of the subject's ability to communicate their choices or preferences, which can include the use of nonverbal means such as eye contact or gestures. The assessment is made on an 8-point Likert scale (0-7) with 0 denoting normal functioning and 7 the most severe impairment.
Measured: 12 Weeks Treatment Duration
Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Verbal Communication (RTT-VCOM)
Clinical assessment of the subject's ability to communicate verbally (e.g. words and phrases). The assessment is made on an 8-point Likert scale (0-7) with 0 denoting normal functioning and 7 the most severe impairment.
Measured: 12 Weeks Treatment Duration
Change From Baseline to Week 12 in Clinical Global Impression-Severity (CGI-S)
A 7 point scale that rates the severity of the subject's illness at the time of assessment, relative to the clinician's experience with subjects who have the same diagnosis. A subject is assessed on severity of illness at the time of rating: 1, normal, not at all ill; 2, borderline ill; 3, mildly ill; 4, moderately ill; 5, markedly ill; 6, severely ill; or 7, extremely ill.
Measured: 12 Weeks Treatment Duration
Change From Baseline to Week 12 in Rett Syndrome Caregiver Burden Inventory (RTT-CBI) Total Score (Items 1-24)
The Rett Syndrome Caregiver Burden Inventory (RTT-CBI) scale is intended to directly address caregiver burden and indirectly assess the significance of treatment effects on function in the context of activities of daily living. Ratings are on a 5-point Likert scale including: 0-never; 1-rarely; 2-sometimes; 3-frequently and 4-nearly always. As in the original Caregiver Burden Inventory, the RTT-CBI has 24 negatively worded items (items 1 through 24) yielding a total score up to 96. The RTT-CBI the range is 0 to 96 and a higher score represents a worse outcome.
Measured: 12 Weeks Treatment Duration
Change From Baseline to Week 12 in Overall Quality of Life Rating of the Impact of Childhood Neurologic Disability Scale (ICND)
The overall quality of life of the subject is also rated by responding to the following: "Please rate your child's overall 'Quality of Life' on the scale below. Choose the number which you feel is best and circle it." The choices range from 1 ("Poor") to 6 ("Excellent").
Measured: 12 Weeks Treatment Duration
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Nov 2019
- Study start6 Nov 2019
- Primary completion28 Oct 2021
- Study completion28 Oct 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Rett SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 187 participants (actual)
Sponsor and investigators
- Lead sponsor
- ACADIA Pharmaceuticals Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04181723
- Sponsor's protocol ID
- ACP-2566-003
- Status last verified
- Apr 2024
- First submitted
- 26 Nov 2019
- Last updated
- 8 Apr 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Darwish M, Passarell J, Youakim JM, Bradley H, Bishop KM. Exposure-Response Efficacy Modeling to Support Trofinetide Dosing in Individuals with Rett Syndrome. Adv Ther. 2024 Apr;41(4):1462-1480. doi: 10.1007/s12325-024-02796-y. Epub 2024 Feb 16.
PubMed 38363467Parent H, Ferranti A, Niswender C. Trofinetide: a pioneering treatment for Rett syndrome. Trends Pharmacol Sci. 2023 Oct;44(10):740-741. doi: 10.1016/j.tips.2023.06.008. Epub 2023 Jul 16.
PubMed 37460385Neul JL, Percy AK, Benke TA, Berry-Kravis EM, Glaze DG, Marsh ED, Lin T, Stankovic S, Bishop KM, Youakim JM. Trofinetide for the treatment of Rett syndrome: a randomized phase 3 study. Nat Med. 2023 Jun;29(6):1468-1475. doi: 10.1038/s41591-023-02398-1. Epub 2023 Jun 8.
PubMed 37291210Neul JL, Percy AK, Benke TA, Berry-Kravis EM, Glaze DG, Peters SU, Jones NE, Youakim JM. Design and outcome measures of LAVENDER, a phase 3 study of trofinetide for Rett syndrome. Contemp Clin Trials. 2022 Mar;114:106704. doi: 10.1016/j.cct.2022.106704. Epub 2022 Feb 8.
PubMed 35149233
Study at a glance
From the registry record, last updated by the sponsor on 8 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome
- ClinicalTrials.gov ID
- NCT04181723
- Acronym / sponsor ID
- ACP-2566-003
- Conditions
- Rett Syndrome
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 187 participants (actual)
- Lead sponsor
- ACADIA Pharmaceuticals Inc.Industry (pharma, biotech, devices)
- Study start
- 6 Nov 2019 (actual)
- Primary completion
- 28 Oct 2021 (actual)
- Study completion
- 28 Oct 2021 (actual)
- First posted
- 29 Nov 2019 (actual)
- Results
- Posted 8 Apr 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To investigate the efficacy of treatment with oral trofinetide versus placebo in females with Rett syndrome
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Drug - Trofinetide | Experimental | Drug: TrofinetideTrofinetide solution of 30-60 mL based on the subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube) |
| Placebo | Placebo comparator | Other: PlaceboTrofinetide placebo solution of 30-60 mL based on the subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube) |
Who may qualify
- Ages
- 5 to 20 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (9 listed)
- Female subjects 5 to 20 years of age, inclusive, at Screening
- Body weight ≥12 kg at Screening
- Can swallow the study medication provided as a liquid solution or can take it by gastrostomy tube
- Has classic/typical Rett syndrome (RTT)
- Has a documented disease-causing mutation in the MECP2 gene
Key exclusion criteria (7 listed)
- Has been treated with insulin within 12 weeks of Baseline
- Has current clinically significant cardiovascular, endocrine (such as hypo- or hyperthyroidism, Type 1 diabetes mellitus, or uncontrolled Type 2 diabetes mellitus), renal, hepatic, respiratory or gastrointestinal disease (such as celiac disease or inflammatory bowel disease) or has major surgery planned during the study
- Has a history of, or current, cerebrovascular disease or brain trauma
- Has significant, uncorrected visual or uncorrected hearing impairment
- Has a history of, or current, malignancy
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TrofinetideTrofinetide solution administered based on the subject's weight at Baseline, twice daily for 12 weeks | Drug | Drug - Trofinetide |
| PlaceboTrofinetide placebo solution administered based on the subject's weight at Baseline, twice daily for 12 weeks | Other | Placebo |
Primary outcome
Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score - Change From Baseline to Week 12
Time frame: Baseline and Week 12
The RSBQ is a 45-item caregiver-completed rating scale that includes 45 items, 39 of them grouped into 8 subscales, whose ratings reflect the severity and frequency of symptoms. Items are rated as 0 (not true), 1 (somewhat or sometimes true), or 2 (very true). The 8 subscales are general mood, breathing problems, hand behavior, face movements, body rocking/expressionless face, night-time behaviors, fear/anxiety, and walking/standing. Scores for item 31 are reversed in the calculation of the total score. The total score ranges from 0 to 90 and is calculated as the sum of the item scores. Higher scores mean worse behaviour.
Clinical Global Impression-Improvement (CGI-I) Score at Week 12
Time frame: 12 Weeks Treatment Duration
To rate how much the subject's illness has improved or worsened relative to a baseline state, a 7-point scale is used from 1=very much improved, 2=much improved, 3=minimally improved, 4=no change, 5=minimally worse, 6=much worse, 7=very much worse.
9 secondary outcomes
Change From Baseline to Week 12 in Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist - Social Composite Score (CSBS-DP-IT Social)
Time frame: 12 Weeks Treatment Duration
Change From Baseline to Week 12 in Impact of Childhood Neurologic Disability Scale (ICND) Total Score
Time frame: 12 Weeks Treatment Duration
Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Hand Function (RTT-HF)
Time frame: 12 Weeks Treatment Duration
Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ambulation and Gross Motor Skills (RTT-AMB)
Time frame: 12 Weeks Treatment Duration
Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ability to Communicate Choices (RTT-COMC)
Time frame: 12 Weeks Treatment Duration
Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Verbal Communication (RTT-VCOM)
Time frame: 12 Weeks Treatment Duration
Change From Baseline to Week 12 in Clinical Global Impression-Severity (CGI-S)
Time frame: 12 Weeks Treatment Duration
Change From Baseline to Week 12 in Rett Syndrome Caregiver Burden Inventory (RTT-CBI) Total Score (Items 1-24)
Time frame: 12 Weeks Treatment Duration
Change From Baseline to Week 12 in Overall Quality of Life Rating of the Impact of Childhood Neurologic Disability Scale (ICND)
Time frame: 12 Weeks Treatment Duration
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Apr 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 38363467Darwish M, Passarell J, Youakim JM, Bradley H, Bishop KM. Exposure-Response Efficacy Modeling to Support Trofinetide Dosing in Individuals with Rett Syndrome. Adv Ther. 2024 Apr;41(4):1462-1480. doi: 10.1007/s12325-024-02796-y. Epub 2024 Feb 16.
- Derived from this study: PubMed 37460385Parent H, Ferranti A, Niswender C. Trofinetide: a pioneering treatment for Rett syndrome. Trends Pharmacol Sci. 2023 Oct;44(10):740-741. doi: 10.1016/j.tips.2023.06.008. Epub 2023 Jul 16.
- Derived from this study: PubMed 37291210Neul JL, Percy AK, Benke TA, Berry-Kravis EM, Glaze DG, Marsh ED, Lin T, Stankovic S, Bishop KM, Youakim JM. Trofinetide for the treatment of Rett syndrome: a randomized phase 3 study. Nat Med. 2023 Jun;29(6):1468-1475. doi: 10.1038/s41591-023-02398-1. Epub 2023 Jun 8.
- Derived from this study: PubMed 35149233Neul JL, Percy AK, Benke TA, Berry-Kravis EM, Glaze DG, Peters SU, Jones NE, Youakim JM. Design and outcome measures of LAVENDER, a phase 3 study of trofinetide for Rett syndrome. Contemp Clin Trials. 2022 Mar;114:106704. doi: 10.1016/j.cct.2022.106704. Epub 2022 Feb 8.
Related trials for Rett Syndrome
RecruitingPhase 3
A Phase 3 Study of Fenfluramine Hydrochloride in Rett Syndrome
Rett Syndrome
Active, not recruiting
Measuring Impact of Computer Gaming on Arm Use in Rett Syndrome
Rett Syndrome
Active, not recruiting
Technology-supported Motor Rehabilitation for People With Rett Syndrome
Rett Syndrome
RecruitingObservational study
Neurophysiologic Biomarkers in Rett Syndrome
Rett Syndrome, RTT
RecruitingPhase 1/2
ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS)
Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
21 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Trofinetide in people with Rett Syndrome, run by ACADIA Pharmaceuticals Inc. at 21 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 21 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 8 Apr 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Rett Syndrome Behaviour Questionnaire (RSBQ) Total Score - Change From Baseline to Week 12 (time frame: Baseline and Week 12). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04181723, which also lists 4 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Apr 2024.